PhaseRx Inc (NASDAQ:PZRX) investors are overwhelmingly excited today after the small biotech announced that its lead candidate, PRX-OTC, which is being developed for the treatment of ornithine transcarbamylase deficiency (OTCD), has received orphan drug designation by the U.S. Food and Drug Administration (FDA).
PhaseRx reacted to the news, sky skyrocketing nearly 100% to $2.28, as of this writing.
“The FDA’s decision to grant PRX-OTC orphan drug designation for OTCD is another important milestone in the development of our lead product candidate, as we prepare to file the IND by the end of 2017 and initiate our clinical trial in 2018,” said Robert W. Overell, Ph.D., president and chief executive officer. “PRX-OTC is the first of three drugs in development using our Hybrid mRNA Technology™, and we believe it has the potential to correct the disease in children, a population that could particularly benefit from treatment for this rare disease. Our team at PhaseRx is driving hard to advance these drugs to help the lives of families affected by this devastating liver disease that causes irreversible brain damage and potentially fatal ammonia toxicity.”
The FDA grants orphan drug designation to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval. (Original Source)
PhaseRx, Inc. engages in the development of novel RNA-based drugs. The firm RNA therapeutics company developing treatments for orphan liver disease. It is utilizing its proprietary hybrid mRNA delivery system, which offers the ability to deliver messenger RNA therapeutics predictably to selected tissues in vivo, thereby unlocking the value of mRNA as a new therapeutic modality.